Tacrolimus Combined With Low-dose Prednisone for Treatment of Myasthenia Gravis

NCT ID: NCT04768465

Last Updated: 2021-02-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

160 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-01-01

Study Completion Date

2024-10-31

Brief Summary

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This study is designed to evaluate the effectiveness and safety of tacrolimus combined with low-dose prednisone in the management of myasthenia gravis patients, compared to tacrolimus as initial immune monotherapy.

Detailed Description

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This is a single center, observational real-world study recruiting myasthenia gravis patients from Neurology Departments of Xuanwu Hospital, aiming to compare effectiveness and safety of 2 different inmunotherapy for MG. The study plans to recruit 160 MG participants and divides into 2 treatment groups according to physician's judgment and preferences of patients, one is combined immunotherapy group in which tacrolimus added with low-dose prednisone (0.25mg/kg/d), and the other is tacrolimus monotherapy group. Both groups can be treated with pyridostigmine to relieve symptoms. Patients are followed up at 1, 3 and 6 month after treatment initiation to assess the efficacy of both regimen. The primary outcome is the change of MG-ADL scores. Also, liver and renal functions are tested to monitor any side effects. Patients' clinical records are uploaded to an online database.

Conditions

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Myasthenia Gravis

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Combined Immunotherapy

MG patients are treated with tacrolimus combined with low-dose prednisone (0.25mg/kg/d).

Symptomatic treatment like pyridostigmine bromide can be added to relieve symptoms (≤480mg/d).

Pyridostigmine, Prednisone, Tacrolimus

Intervention Type DRUG

Dose of tacrolimus should be initiated based on CYP3A5\*3 polymorphism and adjusted to achieve blood trough concentration of 4.8-10.0 ng/ml. Prednisone is administrated with an initial dose of 0.25mg/kg/d and started to tamper with the achievement of MMS or the presence of any intolerable side effects. The rate of tampering is considered by the physician, usually no more than 5mg/month. If the participants failed to maintain MMS, dose of prednisone should be increased 5mg/week to 0.25mg/kg/d and maintained until MMS reached again. After MMS sustained for 1 month, prednisone dose would be tapered again with 2.5mg/month. Calcium and potassium supplements and gastric mucosa protectors could be addressed to avoid any adverse effects of prednisone. Treatment regimens are determined based on the physician's judgment and preferences of the patients. This study was observational and do not change the clinical course of patients.

Tacrolimus monotherapy

MG patients are treated with tacrolimus as initial immune monotherapy. Symptomatic treatment like pyridostigmine bromide can be added to relieve symptoms (≤480mg/d).

Pyridostigmine, Tacrolimus

Intervention Type DRUG

Dose of tacrolimus should be initiated based on CYP3A5\*3 polymorphism and adjusted to achieve blood trough concentration of 4.8-10.0 ng/ml. Treatment regimens are determined based on the physician's judgment and preferences of the patients. This study was observational and do not change the clinical course of patients.

Interventions

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Pyridostigmine, Prednisone, Tacrolimus

Dose of tacrolimus should be initiated based on CYP3A5\*3 polymorphism and adjusted to achieve blood trough concentration of 4.8-10.0 ng/ml. Prednisone is administrated with an initial dose of 0.25mg/kg/d and started to tamper with the achievement of MMS or the presence of any intolerable side effects. The rate of tampering is considered by the physician, usually no more than 5mg/month. If the participants failed to maintain MMS, dose of prednisone should be increased 5mg/week to 0.25mg/kg/d and maintained until MMS reached again. After MMS sustained for 1 month, prednisone dose would be tapered again with 2.5mg/month. Calcium and potassium supplements and gastric mucosa protectors could be addressed to avoid any adverse effects of prednisone. Treatment regimens are determined based on the physician's judgment and preferences of the patients. This study was observational and do not change the clinical course of patients.

Intervention Type DRUG

Pyridostigmine, Tacrolimus

Dose of tacrolimus should be initiated based on CYP3A5\*3 polymorphism and adjusted to achieve blood trough concentration of 4.8-10.0 ng/ml. Treatment regimens are determined based on the physician's judgment and preferences of the patients. This study was observational and do not change the clinical course of patients.

Intervention Type DRUG

Other Intervention Names

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Pyridostigmine Bromide Deltacortone/Meticorten/Prednisone Acetate Tablets Prograf/FK506 Pyridostigmine Bromide Prograf/FK506

Eligibility Criteria

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Inclusion Criteria

* Age ≥18
* Clinical Diagnosis of MG is confirmed based on typical clinical features of fluctuating muscle weakness, with at least 1 of the following supporting evidence:

1. positive clinical response to acetylcholinesterase inhibitor
2. positive AchR-Ab or MuSK-Ab testing
3. decrement \>10% in repetitive nerve stimulations study (RNS) or increased jitter on single-fibre electromyography (SFEMG)
* MGFA clinical classification: I - IV
* Baseline MG-ADL ≥ 3
* Disease course from onset to enrollment ≤ 12 months
* Cooperation to followup
* Written informed consent

Exclusion Criteria

* Initiation of immunosuppressant for MG prior to screening, including Prednisone, Methylprednisolone, Azathioprine, Methotrexate, Cyclosporine A, Mycophenolate Mofetil, Tacrolimus and Cyclophosphamide
* Treatment of immunosuppressant for other concomitant disease 6 months prior to recruitment
* Rapid immunosuppressive treatments like Intravenous immunoglobulin or plasma exchange 1 month prior to recruitment
* Thymectomy within 3 months prior to Screening
* Concomitant chronic degenerative, psychiatric, or neurologic disorder that can cause weakness or fatigue
* Consciousness, dementia or schizophrenia
* Pregnancy or lactation, unwillingness to avoid pregnancy
* Uncontrolled hypertension or diabetes, Liver or kidney dysfunction, Cataract, Severe osteoporosis, Femoral head necrosis; Hyperkalemia, HIV, Acute or chronic infection
* Other conditions that would preclude participation
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Da, Yuwei, M.D.

INDIV

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Yuwei Da, M.D.

Role: STUDY_CHAIR

Xuanwu Hospital, Beijing

Locations

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Yuwei Da

Beijing, Beijing Municipality, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Yuwei Da, M.D.

Role: CONTACT

00-86-010-83198493

Yuwei Da, M.D.

Role: CONTACT

00-86-010-83198493

Facility Contacts

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Yuwei Da, M.D.

Role: primary

00-86-010-83198492

Other Identifiers

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1.0

Identifier Type: -

Identifier Source: org_study_id

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